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Bethesda,
Maryland20892

Purpose:

This study will examine the effectiveness of the drug pirfenidone (Deskar) in improving
heart function in patients with hypertrophic cardiomyopathy (HCM). Stiffening of the heart
muscle in patients with HCM impairs the heart's ability to relax and thus fill and empty
properly. This can lead to heart failure, breathlessness and excessive fatigue. The
heart's inability to relax may be due to scarring, or fibrosis, in the muscle wall. This
study will test whether pirfenidone can reduce fibrosis, improve heart relaxation and reduce
abnormal heart rhythms.
Men and women 20 to 75 years old with HCM may be eligible for this study. Participants will
undergo a physical examination, blood tests, and other tests and procedures, described
below, to assess heart function. When the tests are completed, patients will be randomly
assigned to one of two treatment groups. One group will take a pirfenidone capsule and the
other will take a placebo (a look-alike pill with no active ingredient) twice a day with
meals for 6 months. For the pirfenidone group, the dose of drug will be increased gradually
from 400 to 800 milligrams. At the end of 6 months, all patients will repeat the physical
examination and heart tests that were done before starting medication. These include:
- Electrocardiogram (ECG) - electrodes are attached to the heart to record the heart's
electrical activity, providing information on the heartbeat.
- Echocardiogram - a probe held against the chest wall uses sound waves to produce images
of the heart, providing information on the function of the heart chambers.
- 24-hour Holter monitor - a 24-hour recording of the electrical activity of the heart
monitors for abnormal heartbeats or conduction abnormalities.
- Magnetic resonance imaging (MRI) - Radiowaves and a strong magnetic field are used to
produce images of the heart, providing information on the thickness and movement of the
heart muscle.
- Radionuclide angiogram - a radioactive tracer is injected into a vein and a special
camera is used to scan the heart, providing information on the beating motion of the
heart. Scans are obtained at rest and after exercise.
- Cardiac (heart) catheterization - a catheter (thin plastic tube) is inserted into a
blood vessel in the groin and advanced to the heart to record pressures and take
pictures inside the heart.
- Electrophysiology study - a catheter is inserted into a blood vessel in the groin and
advanced to the heart to record electrical activity, providing information on abnormal
heart rhythms. This procedure is done at the time of the heart catheterization.
- Cardiac biopsy - a catheter is inserted into a blood vessel in the groin and advanced
to the heart to remove a small sample of heart muscle for microscopic examination.
This procedure is done at the end of the heart catheterization.

Study summary:

Left ventricular (LV) diastolic dysfunction and arrhythmias are important causes of
morbidity and mortality in HCM. These abnormalities are believed to be in part due to
myocardial fibrosis which frequently complicates HCM. Several studies indicate that
pirfenidine can safely inhibit progression or cause regression of fibrotic lesions. We
therefore propose to perform a six-month study that examines the ability of pirfenidone to
improve LV diastolic dysfunction, exercise performance, and electrophysiologic abnormalities
in symptomatic patients with severe HCM.